This "orphan disease" will suddenly edema lead to suffocation and death, and "orphan medicine" will be found at the Expo.

Rare diseases are a collective term for many diseases with very low prevalence, also known as "orphan diseases", such as albinism and motor neuron disease.

At this year's Expo, a number of "orphan diseases" found corresponding "orphan drugs".

Hereditary angioedema (HAE) is a rare autosomal dominant disorder in which acute edema occurs unpredictably in multiple parts of the patient's body. Among them, edema in the larynx progresses rapidly, and the average time from edema to asphyxia is 4.6 hours, which can lead to death of asphyxia in severe cases.

According to statistics, 58.9% of HAE patients in China have experienced laryngeal edema, and the fatality rate can reach up to 40%.

Takeda Pharmaceutical's innovative drug Fei Zeyu (Etibant acetate injection) specifically targets the acute onset of HAE and officially benefits Chinese HAE patients at the end of October this year.

At the Expo, Takeda Pharmaceutical also exhibited three drugs for the treatment of rare bleeding diseases, namely hemophilia A, vascular hemophilia, and acquired hemophilia.

Wang Xuan, head of Takeda Pharmaceutical's Asia Development Center, said that in China, Takeda Pharmaceutical expects to market more than 15 innovative drugs within five years, of which more than half are rare disease drugs.

Thanks to the "spillover effect" of the Expo, this year, Takeda Pharmaceutical has approved or listed 4 innovative products for rare diseases, covering many fields such as Fabre disease, Gaucher disease, and hereditary angioedema.

Column editor-in-chief: Chen Xisha Title image Source: Chen Xisha Photo

Source: Author: Chen Xisha